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2016 ; 24
(3
): 447-57
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Engineered Viruses as Genome Editing Devices
#MMPMID26336974
Chen X
; Gonçalves MA
Mol Ther
2016[Mar]; 24
(3
): 447-57
PMID26336974
show ga
Genome editing based on sequence-specific designer nucleases, also known as
programmable nucleases, seeks to modify in a targeted and precise manner the
genetic information content of living cells. Delivering into cells designer
nucleases alone or together with donor DNA templates, which serve as surrogate
homologous recombination (HR) substrates, can result in gene knockouts or gene
knock-ins, respectively. As engineered replication-defective viruses, viral
vectors are having an increasingly important role as delivery vehicles for donor
DNA templates and designer nucleases, namely, zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs) and clustered, regularly
interspaced, short palindromic repeats (CRISPR)-associated Cas9 (CRISPR-Cas9)
nucleases, also known as RNA-guided nucleases (RGNs). We review this dual role
played by engineered viral particles on genome editing while focusing on their
main scaffolds, consisting of lentiviruses, adeno-associated viruses, and
adenoviruses. In addition, the coverage of the growing body of research on the
repurposing of viral vectors as delivery systems for genome editing tools is
complemented with information regarding their main characteristics, pros, and
cons. Finally, this information is framed by a concise description of the chief
principles, tools, and applications of the genome editing field as a whole.